Egetis Therapeutics Ab · OMXSTO:EGTX
Egetis Therapeutics Ab — Investor Presentation (October 2026)
62 slides from Egetis Therapeutics Ab's investor presentation, October 2026
Slide 1: We Care for the Rare – Corporate presentation October 2026
WE CARE FOR THE RARE Corporate presentation October 2026 Emcitate® (tiratricol) US FDA Approval on September 28, 2026 EU Approval and Launch in Germany in May 2025 For investors and analysts only ©2026 Egetis Therapeutics. All rights reserved. 1
Slide 2: Disclaimer
Disclaimer This presentation contains forward-looking statements within the meaning of applicable securities laws, including the commercialization and launch of Emcitate in the United States and Europe; anticipated pricing, revenue, and market opportunity; the expected capabilities of the Company's
Slide 3: Egetis Overview
WE CARE FOR THE RARE Egetis Overview An integrated orphan drug company, focusing on late-stage development and commercialization
Slide 4: Strategic Value Drivers
Strategic Value Drivers Changing Patients’ and Families’ Lives 1 Emcitate addresses a devastating rare disease with severe lifelong morbidity, shortened life expectancy and substantial caregiver burden, consistent with Egetis’ mission to bring unique therapies that improve and extend patient lives U
Slide 5: Strong Regulatory Status As An Orphan Drug Commercial Stage Company
Egetis Maintains Strong Regulatory Status As An Orphan Drug Commercial Stage Company Key Regulatory & Commercial Highlights First & Only FDA- Breakthrough Fast Track Rare Pediatric Approved Drug Therapy Designation Designation Disease Designation For MCT8 Deficiency; Granted by the FDA Granted by th
Slide 6: Strong Execution in 2025-26 Positions Egetis for Emcitate US Launch
Strong Execution in 2025-26 Positions Egetis for Emcitate US Launch in 2026 • Japan – development plan alignment with PMDA EC approves Emcitate Positive Phase 3 • Completion and acceptance • Launch of Emcitate FDA Breakthrough in the US as first and only ReTRIACt of NDA Therapy treatment for MCT8 wi
Slide 7: MCT8 Deficiency Results in Dysfunctional Thyroid Hormone Trafficking
MCT8 Deficiency Results in Dysfunctional Thyroid Hormone Trafficking MCT8 Deficiency Key Features T4 10 Months 35 Years 1 in 70K T3 Estimated Incidence Median Age of Diagnosis Median Life Expectancy (Male Births) TSH ~30% Sudden Cardiac 90% Death Hypotonia, Hypertonia & Patients Dying Persistence of
Slide 8: Emcitate Mechanism of Action in MCT8 Deficiency
Emcitate® (tiratricol) Mechanism of Action in MCT8 Deficiency with clear scientific and mechanistic rationale • Tiratricol is a small molecule, thyroid hormone receptor agonist • Unlike T3, tiratricol can cross cellular membranes without a functional MCT8 transporter • Tiratricol can bypass the prob
Slide 9: Emcitate Approved in EU and US
Emcitate® (tiratricol) Approved in EU and US Robust dataset in an ultra-rare genetic disease 3 clinical studies 3 sources of real-world evidence EMC Cohort EMC Survival US Expanded ReTRIACt Triac Trial I Triac Trial II Study Study Access Program N=20(1) N=46 N=22 N=67 N>600 N=30(2) Randomized placeb
Slide 10: Tiratricol treatment associated with survival benefits
Tiratricol treatment in patients with MCT8 deficiency has been shown to be associated with survival benefits In the Triac Trial I, tiratricol reached target level serum T3 & improvements Tiratricol has been shown to be in clinically relevant outcome measures associated with a 3x lower risk of mortal
Slide 11: Tiratricol's Effect on T3 Lowering May Explain Mortality Benefit
Tiratricol’s Effect on T3 Lowering May Explain Mortality Benefit Emcitate Source for evidence of (tiratricol) efficacy T3 Triac Trial I, Triac Trial II, ReTRIACt, EMC Cohort Study Triac Trial I, Immune Body weight HR BP Arrhythmia EMC Cohort system Study RPP Triac Trial I, ReTRIACt Infection Sudden
Slide 12: Final USPI for Emcitate
Final USPI for Emcitate Approved by the FDA on September 28th EU-Style Indication Statement 1 Treatment of peripheral thyrotoxicosis in adults and pediatric patients with MCT8 deficiency No Age Restriction 2 Adults and pediatric patients alike Narrow Limitation Of Use 3 LoU for primary hypothyroidis
Slide 13: Consistent MCT8 Deficiency Incidence Estimates
Multiple Sources Lead to Consistent MCT8 Deficiency Incidence Estimates Available Data Leads to Consistent Supporting Our Relevant Sources & Data MCT8 Deficiency Incidence Estimates Conservative Estimate • Multiple cohorts of patients with X- linked mental retardation under study Visser et al., Clin
Slide 14: Emcitate Launch By Egetis and Partners
Emcitate® (tiratricol) Launch By Egetis and Partners Executing the US & European market preparations and launches through the Egetis team To optimize for launch, Egetis is focusing in-house resources on the US and Europe while expanding to additional countries through partners Japan license deal wit
Slide 15: A First for the U.S. MCT8 Deficiency Community
A First for the U.S. MCT8 Deficiency Community FIRST AND ONLY FDA-approved treatment for peripheral thyrotoxicosis in adults and pediatric patients with MCT8 deficiency in the U.S. Not recommended for the treatment of primary hypothyroidism. First approved therapy Egetis’ first U.S. commercial produ
Slide 16: US Launch Readiness: Building Momentum Toward Launch
16 US Launch Readiness: Building Momentum Toward Launch Launch readiness remains on track, with a fully assembled team and activated infrastructure Key Priorities Functional Readiness Regulatory / Medical Affairs Commercial & Field NDA 1 Execute EAP continuity of care readiness ✓ FDA approval 28 Sep
Slide 17: US Launch Readiness: Continuity of Care for EAP Patients
U.S. Launch Readiness: Continuity of Care for EAP Patients is Our Top Priority Tiratricol EAP continuity of care — ensuring Patient-level transition planning uninterrupted treatment Confirm the pathway and treating physician for each EAP patient at approval Enrollment & prescriber readiness A coordi
Slide 18: A Concentrated Starting Point Supports Focused Launch Execution
A Concentrated Starting Point Supports Focused Launch Execution 60+ Patients program treated in the EAP Patients concentrated EAP patients managed at a few experienced centers 17 Sites Physicians engaged Every EAP investigator and care team engaged multiple times ahead of approval Ready to execute A
Slide 19: Egetis' Pricing Principles
Egetis’ Pricing Principles Affordability Value of Our Access to Therapy Support appropriate patient Medicine Sustaining Innovation Enable broad, equitable and access and help eligible Reflect the high burden and Fund continued research in reliable access to therapy for patients & families navigate u
Slide 20: Egetis RareLink: One Connected Patient Support Program
Egetis RareLink : One Connected Patient Support Program Patient Support Care Manager Liaison Help coordinate Personalized treatment insurance requirements education and program and financial support support options Developed in partnership with Educational Specialty Pharmacy PANTHERx Rare Pharmacy M
Slide 21: Active Launch Execution Ahead of Commercial Availability
Active Launch Execution Ahead of Commercial Availability Current Activity Long-Term Value Physicians Disciplined execution Readiness and continuity of care planning Delivery and appropriate patient access Payers Standard of care Commercial and Medicaid coverage EMCITATE for peripheral thyrotoxicosis
Slide 22: Multi-Channel Patient Identification Strategy
Multi-Channel Patient Identification Strategy Analytics & Genetic Testing Partnerships Prospective patient ID partnership with Invitae and Gene Dx is live, flagging every newly confirmed MCTS8 Dx patient directly to Egetis Targeted HCP & Referral Network Engagement Expand field resources in places m
Slide 23: ETA Recommends Emcitate as Long-Term Therapy for MCT8 Deficiency
European Thyroid Association (ETA) Recommends Emcitate (tiratricol) as Long-Term Therapy for MCT8 Deficiency ETA recommends the use of tiratricol as long-term therapy for all patients with MCT8 deficiency, and for certain patients with RTHβ Inaugural 2024 Guidelines were commissioned by the Executiv
Slide 24: A Phased EU Launch Through In-House Commercial Organization
A Phased EU Launch Through In-House Commercial Organization Started in Germany In May 2025 Launch execution in two waves, starting with EU4 Pricing & reimbursement processes Deliver the Emcitate clinical and economic value proposition in P&R processes, outlining: Wave 1 • MCT8 deficiency and its rar
Slide 25: Europe & International
Europe & International Q2 revenue MSEK 17.4, +23% (CER) vs Q2 2025 and +30% vs Q1 2026; finalized national pricing and reimbursement process in Germany and submitted in Spain Germany • Price negotiations with GKV-Spitzenverband concluded in Q2 2026 • Continued disease awareness, physician engagement
Slide 26: Pipeline Overview
Pipeline Overview Emcitate® (tiratricol) – Launched in Germany May 2025; US PDUFA date September 28, 2026 Candidate Preclinical Phase I Phase II / III MAA / NDA Comments Emcitate EU EC approval received Feb 12, 2025 Launched Launched in Germany May 1, 2025 MCT8 deficiency Emcitate US FDA approved Se
Slide 27: RTHβ Disrupts Thyroid Hormone Homeostasis
RTHβ Disrupts Thyroid Hormone Homeostasis, Leading to Imbalanced TH Signalling Between Compartments RTHβ Disrupts Thyroid Hormone Homeostasis, Leading to Imbalanced TH Signalling Between Compartments Treatment resistant ADHD RTHβ Key Features Disrupted TH Impaired hearing and color vision homeostasi
Slide 28: RTHβ: Emcitate Uniquely Positioned to Address High Unmet Need
RTHβ: Emcitate is Uniquely Positioned to Address High Unmet Need Potential indication expansion for Emcitate into larger non-overlapping patient population Emcitate as Potential Treatment for RTHβ • Emcitate efficacious in restoring signaling in majority of TRβ mutations in vitro due to greater affi
Slide 29: RTHβ: Thyroid Hormone Concentration on Triac Treatment
RTHβ: Thyroid Hormone Concentration on Triac Treatment Increased Mortality in RTH𝛽(1) Free T4 Concentration(2) (Untreated RTHβ Patients vs. Control Group) (Emcitate-Treated RTHβ Patients) fT4 Baseline to Nadir 60 A1 A2 50 A3 A4 A5 40 A6 fT4 (pmol/L) A7 30 A8 20 10 0 Baseline 1 Nadir2 Welsh cohort…
Slide 30: RTHβ: TPP & CDP – Where We Stand
RTHβ: TPP & CDP – Where We Stand May Jun-Jul Jul-Aug Sep 5 Sep / Oct Q4/Q1 2027 End Aug Finalize TPP/CDP Project Team WoW TPP & CDP KOL Calls: Rees, KOL Ad Board Regulatory TPP v1.1 + CDP v 1.0 Integrate KOL, Defined workstreams live Chatterjee, Moran, (Porto, ETA) Interactions advisory-board and Pe
Slide 31: RTHβ: Target Product Profile v1.0 – Progress by Attribute
RTHβ: Target Product Profile v1.0 – Progress by Attribute Six Attributes Target / Optimal Profile Population & Indication All ages incl. pediatric; treatment of RTHβ indication (disease modification) Efficacy & Claim FT4 normalization + improvement in clinical manifestations (CV, PRO + disease marke
Slide 32: RTHβ: Emerging Pivotal Trial Design
RTHβ: Emerging Pivotal Trial Design Primary Endpoint - FT4 normalization Biochemical anchor; goal is normalization + supportive clinical improvement Trial Design Population Dosing Safety & Monitoring • Parallel-group, robust • Genetic confirmation • Two pragmatic start doses at • Cardiac: HR, rhythm
Slide 33: Strong Financial Foundation for Strategic Execution
Strong Financial Foundation for Strategic Execution Solid Cash Position Top 10 Largest Shareholders • Cash position as of June 30, 2026: SEK 378 million – Does not reflect net proceeds from expected sale of US Rare Pediatric Disease Priority Review Voucher related to Emcitate • Number of outstanding
Slide 34: Financial Overview – Second Quarter 2026
Financial Overview – Second Quarter 2026 • Total revenue ‒ Q2-2026 of 17.4 MSEK vs. 14.5 MSEK for Q2-2025, +23% YoY CER. • Cost of goods sold impacted by intangible R&D depreciation ‒ During Q2-2026 R&D depreciation of 10.1 MSEK is included in COGS ‒ Excluding the depreciation Gross profit would hav
Slide 35: Patent Granted for MCT8 Deficiency Composition in the U.S.
Egetis Announces Grant of Patent for MCT8 Deficiency Composition in the U.S. • USPTO granted patent PRESS • Provides protection for a novel composition containing tiratricol as active ingredient • Claims cover a method of treating MCT8 deficiency, including dose regiments • Orange Book-listable with
Slide 36: Appendix 1: Overview of MCT8 deficiency
Appendix 1 Overview of MCT8 deficiency 36
Slide 37: MCT8 deficiency results in dysfunctional thyroid hormone trafficking
MCT8 deficiency results in dysfunctional thyroid hormone trafficking MCT8 deficiency has two co-manifestations MCT8 deficiency results in simultaneous too high and too low New Research Sheds Light on Thyroid Hormone Transport thyroid hormone levels – causing system wide issues • In 2003, MCT8 was id
Slide 38: MCT8 deficiency: a detrimental condition with significant unmet medical need
MCT8 deficiency: a detrimental condition with significant unmet medical need What is MCT8 deficiency? What does it mean? What are the challenges? How do you manage the Quick facts from natural history2 disease? • Genetic X-linked disorder ● Non-functional MCT8 protein: ● Patients appear normal at bi
Slide 39: Natural history study revealed poor survival
Natural history study revealed poor survival with a high prevalence of treatable underlying risk factors An international, retrospective, multicentre cohort study from 2014-2020 in 151 patients • 151 patients were enrolled with 73 different MCT8 (SLC16A2) mutations • Median age at diagnosis was 24.0
Slide 40: Appendix 2: Emcitate mode of action and clinical experience
Appendix 2 Emcitate (tiratricol): Mode of action and clinical experience
Slide 41: Discovery of Emcitate (Triac, tiratricol)
Discovery of Emcitate (Triac, tiratricol) T3 Triac (tiratricol) 41 © 2026 Egetis Therapeutics. All rights reserved.
Slide 42: Overview of completed Phase IIb – Triac Trial I
Overview of completed Phase IIb – Triac Trial I • Evaluate the efficacy and safety of oral administration of tiratricol in male patients with MCT8 deficiency of all ages Primary objective and • Highly significant primary outcome - Change in T3 serum concentrations results • Safe and tolerable • Resu
Slide 43: Consistent, clinically relevant and highly significant results
Consistent, clinically relevant and highly significant results Triac Trial I: Reached target level serum T3 & improvements in clinically relevant outcome measures 10 T0 (Baseline) 8 T12 (12 month) p < 0.0001 Serum T3 (nmol/l) 6 4 2 Normal range 0 Endpoints Baseline mean (± SD) 12 months mean (± SD)
Slide 44: Triac Trial I: Positive effect on neurocognitive development
Triac Trial I: Indication of positive effect on neurocognitive development Triac Trial II did not meet its primary endpoints 40 T0 (Baseline) T12 (12 months) 30 GMFM score (%)1) 20 10 0 # # # Patients 1.5 to <4 years 4 to <10 years ≥10 years Age at baseline Source: Groeneweg et al; Lancet D&E 2019 4
Slide 45: Real-world evidence: Long-term efficacy and safety of Emcitate
Real-world evidence: Long-term efficacy and safety of Emcitate® in MCT8 deficiency patients Published in October, 2021 • Investigator-initiated real-world cohort study at 33 sites conducted by the Erasmus Medical Center • Investigated efficacy and safety of Emcitate in 67 patients with MCT8 deficien
Slide 46: New patient cohort of equal size to the Triac Trial I
New patient cohort of equal size to the Triac Trial I Long term follow up, up to >6 years Triac Trial I N=46 Triac Trial I patients New patients Available for long-term From managed access program monitoring N=40 N=27 EMC cohort Published in JCEM N=67 46 © 2026 Egetis Therapeutics. All rights reserv
Slide 47: New cohort confirms primary endpoint results in Triac Trial I
New cohort confirms primary endpoint results in Triac Trial I Fast and durable normalization of T3 values in almost all patients p<0.0001 47 © 2026 Egetis Therapeutics. All rights reserved.
Slide 48: Consistent, clinically relevant and highly significant results across endpoints
Consistent, clinically relevant and highly significant results across endpoints • Data confirm the positive results from previous study, Triac Trial I • Normalization of serum T3 corresponds to improvement in thyroid hormone status in end target tissues • Beneficial effects are maintained or continu
Slide 49: Triac Trial II objective and design
Triac Trial II objective and design: Triac Trial II was designed to investigate a potential additional benefit on neurocognitive development in 22 patients with MCT8 deficiency below 30 months of age treated with Emcitate® (tiratricol) during 96 weeks • Confirm findings from Triac Trial I in younges
Slide 50: Triac Trial II Summary
Triac Trial II Summary • Triac Trial II results: • The numerical improvements versus baseline observed on the primary endpoints of neurocognitive development assessed by the GMFM-88 and BSID-III scales did not show a statistically significant improvement versus historical controls. • The trial confi
Slide 51: ReTRIACt Trial Designed to Support NDA Submission
ReTRIACt Trial Designed to Support NDA Submission Aligned with FDA to Include ReTRIACt Data as Complementary and Not Pivotal • A 30-day, randomized placebo-controlled Patients already on therapy withdrawal study in 15 patients tiratricol tiratricol tiratricol • The study allowed inclusion of patient
Slide 52: ReTRIACt study: Results on primary endpoints
ReTRIACt study: Results on primary endpoints Largest increase R R Patients ordered by change in serum T3 (nmol/L) R during randomized treatment period* R Placebo range [0.49 : 2.08] No change Tiratricol range D [-0.24 : 0.40] Largest decrease * = change from baseline to rescue or end of randomized t
Slide 53: Overview of changes in clinical variables during Triac Trial I
Overview of changes in clinical variables during Triac Trial I 53 T3 within normal range T3 outside normal range © 2026 Egetis Therapeutics. All rights reserved.
Slide 54: Tiratricol treatment associated with survival benefits
Real-world evidence: Tiratricol (Emcitate®) treatment in patients with MCT8 deficiency is associated with survival benefits PRESS • Abstract published ahead of the ETA/ITC Annual Meeting report that treatment with tiratricol (Emcitate®) in patients with MCT8 deficiency is associated with a 3x lower
Slide 55: Post-hoc analysis on patient-centered outcome measures
Post-hoc analysis reports effects of tiratricol on patient-centered outcome measures in patients with MCT8 deficiency • According to the Abstract, there were PRESS improvements upon tiratricol treatment reported by caregivers related to improved interaction (22/39), greater alertness (19/39), improv
Slide 56: Appendix 3: Priority Review Voucher
Appendix 3 Priority Review Voucher
Slide 57: Egetis Granted PRV following U.S. Approval of EMCITATE
Egetis Granted PRV following U.S. Approval of EMCITATE® Recent U.S. Rare Pediatric Disease Voucher (PRV) Value Range: $180M-$220M Overview of PRV Examples of PRVs sold in 2026 • The FDA grants Rare Pediatric Disease designation (RPD) Seller Buyer Value to therapies for serious or life-threatening di
Slide 58: Appendix 4: Leadership Team and Board of Directors
Appendix 4 Leadership Team and Board of Directors
Slide 59: Leadership team with global experience & proven track record
Leadership team with global experience & proven track record Nicklas Westerholm Tiago Nunes, MD, PhD Sofie Broberg CEO Chief Medical Officer VP Regulatory Affairs • Joined 2017 • Joined 2026 • Joined 2026 • AstraZeneca 1995-2017 • SVP, Clinical Development, Mirum Pharmaceuticals • Regulatory affairs
Slide 60: Board of directors
Board of directors Mats Blom Margarida Duarte Chair of the board since 2024 Board member since 2025 • Shares in Egetis: 3,499,762 • Shares in Egetis: 0 • BA, Business Administration & Economics, Lund University; • BS in Pharmaceutical Sciences & Executive Master’s degree MBA, IESE University of Nava
Slide 61: Step-wise building team to execute on key activities for launch
Step-wise building team to execute on key activities at the right time for launch success Key projects driven by recognized industry talents recruited to the Egetis Commercial & Medical Affairs Team – Core team brings launch skills and best practices from in total 150+ years at international compani
Slide 62: Thank you!
Thank you! Egetis Therapeutics egetis.com ir@egetis.com 62
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